Arikayce (amikacin liposome inhalation suspension) / Insmed - LARVOL DELTA

FDA Grants Priority Review to Insmed's sNDA for ARIKAYCE (amikacin liposome inhalation suspension) for Treatment of MAC Lung Disease With PDUFA Target Action Date Set for January 28, 2027 (PRNewswire) - "In its communication to Insmed, the FDA granted Priority Review to the sNDA and set a target action date of January 28, 2027, under the Prescription Drug User Fee Act (PDUFA)....The sNDA for full approval is supported by results from the Phase 3b ENCORE study, the pivotal study conducted to fulfill the post-marketing requirement associated with ARIKAYCE's accelerated approval."

FDA filing • PDUFA • Priority review • Immunology • Infectious Disease • Nontuberculous Mycobacterial Disease • Respiratory Diseases

Insmed announced that five abstracts highlighting data from its respiratory portfolio have been accepted for presentation at the European Respiratory Society (ERS) Congress 2026, taking place Sept. 5–9, 2026, in Barcelona (Insmed Press Release) - "A late-breaking abstract from the Phase 3b ENCORE study will present data evaluating 12 months of treatment with ARIKAYCE plus multidrug therapy in patients diagnosed with a new occurrence of Mycobacterium avium complex (MAC) lung infection who had not received antibiotics...The presentation, titled ‘Microbiologic Outcomes and Recurrence in Patients With Newly Diagnosed Mycobacterium avium Complex Lung Disease (MACLD) in the Phase 3b ENCORE Trial,’ is scheduled for Sept. 6, 2026...Insmed stated the findings address rates of recurrence following treatment for NTM lung disease."

P3 data • Immunology • Infectious Disease • Nontuberculous Mycobacterial Disease • Respiratory Diseases

Understanding the molecular insights of marketed liposomal drugs using molecular dynamics simulations of reduced scale coarse-grained models. (PubMed, NPJ Drug Discov) - "In this study, we employed coarse-grained molecular dynamics simulation to investigate the structural details of the commercially available nine liposomal formulations, with five passive loading systems (Arikayce Kit®, Visudyne®, Ambisome®, Mepact®, and Exparel®) and four active loading systems (Doxil®, Myocet®, Onivyde®, and Vyxeos®)...In four active loading systems, the presence of ions within the liposome cavity promotes the self-aggregation of drugs, thereby enhancing drug retention and release profiles. This study provided valuable molecular insights for marketed liposomes formulation."

Journal

A Simultaneous Assessment of Efficacy and Safety of Amikacin Liposome Inhalation Suspension for Refractory Mycobacterium avium Complex Lung Disease. (PubMed, Clin Ther) - "In this post hoc analysis of CONVERT, patients with refractory MACLD treated with ALIS plus MDT spent more time experiencing positive treatment outcomes compared with patients treated with MDT alone."

Journal • Pulmonary Disease • Respiratory Diseases

Amikacin liposome inhalation suspension in newly diagnosed Mycobacterium avium complex lung disease (ARISE): a 6-month double-blind, active comparator trial. (PubMed, Ann Am Thorac Soc) - P3 | "More patients with newly diagnosed MACLD receiving 6 months of ALIS alongside a macrolide-based regimen achieved culture conversion by month 6 and month 7 numerically more rapidly versus comparator. No new safety signals were identified.Clinical trial registered with www.clinicaltrials.gov (NCT04677543)."

Clinical • Journal • Bronchiectasis • Fatigue • Infectious Disease • Nontuberculous Mycobacterial Disease • Pulmonary Disease • Respiratory Diseases

Amikacin Liposome Inhalation Suspension for Newly Diagnosed Mycobacterium Avium Complex Lung Disease: Efficacy and Safety From a Phase 3b Study (ENCORE) (ATS 2026) - No abstract available

Clinical • Late-breaking abstract • P3 data • Pulmonary Disease • Respiratory Diseases

ENCORE: Study to Evaluate ALIS (Amikacin Liposome Inhalation Suspension) in Participants With Nontuberculous Mycobacterial Lung Infection Caused by Mycobacterium Avium Complex (clinicaltrials.gov) - P3 | N=425 | Completed | Sponsor: Insmed Incorporated | Active, not recruiting ➔ Completed

Trial completion • Infectious Disease • Nontuberculous Mycobacterial Disease • Respiratory Diseases

Liposomal antimicrobials in the fight against bacterial and fungal pathogens: Clinical successes and development challenges. (PubMed, Int J Pharm ) - "Liposomal formulations (spherical vesicles composed of lipid bilayers) have demonstrated remarkable clinical potential in addressing these concerns, as evidenced by the marketed products AmBisome® and Arikayce®. These products, which deliver amphotericin B via parenteral injection and amikacin via inhalation, exemplify how liposomes effectively mitigate drug-associated toxicity, enhance therapeutic efficacy, and overcome the biological barriers inherent to infection sites, including complex microbial biofilms, mucosal interfaces, or the blood-brain barrier...This review emphasises the need for a paradigm shift within liposomal antimicrobial development, encouraging progression from initial research and development toward scalable, reproducible, and economically viable commercial manufacturing platforms. This transition is essential not only for ensuring the global accessibility and affordability of existing therapies but also for expanding the development of..."

Journal • Review • Infectious Disease • Oncology

Insmed stock rises after strong BRINSUPRI launch, ARIKAYCE revenue beat (Investing.com) - "The biopharmaceutical company reported unaudited BRINSUPRI (brensocatib) revenue of approximately $144.6 million for its first full quarter on the market, with full-year 2025 sales reaching approximately $172.7 million. ARIKAYCE (amikacin liposome inhalation suspension) exceeded the company’s guidance range with unaudited global revenues of approximately $433.8 million for 2025, representing 19% YoY growth."

Sales • Immunology • Infectious Disease • Non‐Cystic Fibrosis Bronchiectasis

AKAPI: Amikacin Liposome Inhalation Suspension for Treatment of Mycobacterium Xenopi Pulmonary Infection (clinicaltrials.gov) - P2 | N=190 | Recruiting | Sponsor: Centre Hospitalier Universitaire, Amiens | Not yet recruiting ➔ Recruiting

Enrollment open • Infectious Disease • Pulmonary Disease • Respiratory Diseases

Healthcare Resource Utilization in Refractory MACLD: Comparison of an Amikacin Liposome Inhalation Suspension (ALIS) Cohort with a Non-ALIS Cohort. (PubMed, Pulm Ther) - "Add-on treatment with ALIS in refractory MACLD may lead to reductions in hospitalizations over time and lower odds of hospitalizations compared with add-on treatment with non-ALIS antibiotics."

HEOR • Journal • Nontuberculous Mycobacterial Disease • Pulmonary Disease • Respiratory Diseases

Management and Resolution of Hypersensitivity Pneumonitis-Related Events in Japanese Patients Treated with Amikacin Liposome Inhalation Suspension in the CONVERT and INS-312 Clinical Trials. (PubMed, Infect Dis Ther) - P3 | "At the time of diagnosis, one patient had ground-glass opacity, and the other patient had consolidation in the right lower lobe of the lung; both patients had serum KL-6 levels > 600 U/mL. Changes in chest radiography and KL-6 levels at the onset of respiratory symptoms during ALIS treatment suggest that these assessments may help guide management strategies for potential hypersensitivity pneumonitis in patients taking ALIS.Clinical Trial Registration: NCT02344004 (CONVERT) and NCT02628600 (INS-312)."

Journal • Immunology • Inflammation • Interstitial Lung Disease • Nontuberculous Mycobacterial Disease • Pneumonia • Pulmonary Disease • Respiratory Diseases • Tuberculosis

Assuming successful results from the ENCORE trial, Insmed plans to submit a supplementary new drug application (sNDA) to the U.S. Food and Drug Administration (FDA) for ARIKAYCE in all patients with MAC lung disease in the U.S. in the second half of 2026. (PRNewswire)

FDA filing • Respiratory Diseases

Persistent use of amikacin liposome inhalation suspension associated with lower risk of hospitalizations and emergency room visits in refractory Mycobacterium avium complex lung disease. (PubMed, Respir Med) - "Persistent use of ALIS during the first 12 months of treatment was associated with a lower risk of hospitalizations and ER visits."

Journal • Nontuberculous Mycobacterial Disease • Pulmonary Disease • Respiratory Diseases • Tuberculosis

Natural history and burden of refractory Mycobacterium avium complex pulmonary disease: insights from the US Bronchiectasis and Nontuberculous Mycobacterial Research Registry. (PubMed, Respir Med) - "MACPD imposes substantial burden on patients in terms of symptoms, exacerbations, and hospitalizations. Disease burden was present among refractory patients, at pre-refractory and refractory visits ≥1 year later, and non-refractory patients. Understanding patient characteristics at time of treatment may help timely identification and management of refractory MACPD."

Journal • Retrospective data • Bronchiectasis • Chronic Cough • Cough • Nontuberculous Mycobacterial Disease • Pulmonary Disease • Respiratory Diseases

Pulmonary surfactant-based pirfenidone-loaded nanovesicles for inhalation therapy of idiopathic pulmonary fibrosis. (PubMed, J Control Release) - "When inhaled in mice using a nebulizer, the PFD-PSNVs remained in lung tissues for 24 h, whereas Arikayce, an FDA-approved liposomal formulation for inhalation, was eliminated within 6 h. In a bleomycin sulfate-induced IPF mouse model, inhalation treatment with PFD-PSNVs significantly reduced collagen deposition (76.2 ± 4.1 %, p < 0.01) and α-SMA expression (60.8 ± 3.7 %, p < 0.05) compared with inhalation treatment with the PFD-loaded CtrlNV (PFD-CNVs) formulation and oral administration of PFD. These results indicate that PSNVs have great potential as an inhaled drug delivery system for the treatment of IPF."

Journal • Fibrosis • Idiopathic Pulmonary Fibrosis • Immunology • Pulmonary Disease • Respiratory Diseases • SMAD2 • TGFB1

Longitudinal Changes in St. George's Respiratory Questionnaire Scores Following Culture Conversion in Patients With Mycobacterium Avium Complex Lung Disease (ATS 2025) - P3 | "This abstract is funded by: Insmed Incorporated Rationale Amikacin liposome inhalation suspension (ALIS) is indicated for treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients who do not achieve negative sputum cultures after at least 6 consecutive months of multidrug antibiotic therapy...The same trend was observed across the 3 health domains measured by the SGRQ, with a 7.8 point reduction in Symptoms, 6.2 point reduction in Activity, and 7.9 point reduction in Impact at EOT+3 months (P<0.05 for all). Conclusions After treatment was completed, patients who achieved sputum culture conversion experienced significant reduction in SGRQ scores from baseline, which may indicate improvement across all included health domains."

Pulmonary Disease • Respiratory Diseases

Longitudinal Health Status Improvements Following Culture Conversion in Patients With Mycobacterium Avium Complex Lung Disease (ATS 2025) - P3 | "This abstract is funded by: Insmed Incorporated Rationale Amikacin liposome inhalation suspension (ALIS) is indicated for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients who do not achieve negative sputum cultures after a minimum of 6 consecutive months of multidrug therapy...At EOT+3 months, the index score improved significantly by 0.075 points from baseline (P=0.018) where 33% of patients had an improvement exceeding the MCID. Conclusions Patients achieving culture conversion experienced significant improvements in HRQoL as measured by EQ-VAS and EQ-5D-3L scores, particularly after treatment ended, with almost 50% of patients reaching or exceeding the MCID threshold for the EQ-VAS."

Pulmonary Disease • Respiratory Diseases

Healthcare Resource Utilization Associated With Treatment for Refractory Mycobacterium Avium Complex Lung Disease: Comparison of Amikacin Liposome Inhalation Suspension (ALIS) vs Non-ALIS Antibiotic Treatment (ATS 2025) - "As no diagnosis codes for MACLD exist in claims, we defined 2 cohorts of patients receiving treatment for possible rMACLD as follows: 1) patients who initiated ≥28 days of continuous treatment with ALIS; and 2) patients who initiated ≥28 days of continuous treatment with ≥1 non-ALIS antibiotic used to treat rMACLD (i.e., parenteral aminoglycoside, clofazimine, fluoroquinolones, linezolid, tedizolid, or bedaquiline) ≥6 months after initiating treatment used for MACLD (i.e., with a combination of ≥2 antibiotics from a macrolide, ethambutol, and a rifamycin)...No statistically significant changes from baseline in ER visits were observed in either group. Conclusions Patients treated with ALIS for rMACLD had statistically significant reductions in hospitalizations from baseline during the 7-12 month post-index period, whereas no statistically significant improvements in hospitalizations were observed in the non-ALIS antibiotics group."

HEOR • Nontuberculous Mycobacterial Disease • Pulmonary Disease • Respiratory Diseases

Recent Progress and Anticipated Milestones by Program (PRNewswire) - "ARIKAYCE global revenue grew 23% in the first quarter of 2025 compared to the first quarter of 2024, reflecting double-digit year-over-year growth in the U.S., Japan, and Europe; The Company anticipates the topline readout of the Phase 3 ENCORE trial in the first half of 2026 in patients with newly diagnosed or recurrent Mycobacterium avium complex (MAC) lung disease who have not started antibiotics, which will include the change from baseline in respiratory symptom score at Month 13 and the percentage of patients achieving durable culture conversion at Month 15; The Company plans to submit a supplementary new drug application (sNDA) to the U.S. Food and Drug Administration (FDA) for ARIKAYCE in all patients with MAC lung disease in the U.S. in the second half of 2026....Cost of product revenues (excluding amortization of intangibles) was $21.3 million for the first quarter of 2025, compared to $17.5 million for the first quarter of 2024."

FDA filing • P3 data • Sales • Infectious Disease • Respiratory Diseases

Trial Conduct, Baseline Characteristics, and Symptom Burden of Patients in the ARISE Study. (PubMed, Pulm Ther) - P3 | "Patients in ARISE were representative of a real-world patient population with MACLD. Comorbid chronic respiratory diseases were common in patients with new or recurrent MACLD, and substantial disease burden at the time physicians initiated MACLD treatment was evidenced by impairment across measures of fatigue and QOL-B domains."

Journal • Asthma • Bronchiectasis • Chronic Obstructive Pulmonary Disease • Fatigue • Immunology • Infectious Disease • Nontuberculous Mycobacterial Disease • Pulmonary Disease • Respiratory Diseases

Insmed Reports Fourth-Quarter and Full-Year 2024 Financial Results and Provides Business Update (PRNewswire) - "ARIKAYCE global revenue grew 19% in 2024 compared to 2023...The Company continues to anticipate a topline readout for ENCORE in the first quarter of 2026, with the submission of a supplementary new drug application (sNDA) to the FDA for ARIKAYCE in all patients with MAC lung disease in the U.S. expected later in 2026...Regulatory submissions for brensocatib in the EU, UK, and Japan are planned for 2025, with commercial launches anticipated in 2026...The Phase 2b BiRCh study of brensocatib in patients with chronic rhinosinusitis without nasal polyps (CRSsNP) remains on track to report topline results by the end of 2025...The Company plans to initiate a Phase 3 study of TPIP in patients with PH-ILD in the second half of 2025. Enrollment in the Phase 2b study of TPIP in pulmonary arterial hypertension (PAH) completed in December 2024...Insmed continues to anticipate topline data from the study in the middle of 2025."

Commercial • Filing • New P3 trial • P2b data • P3 data: top line • Infectious Disease • Nasal Polyps • Pulmonary Arterial Hypertension • Pulmonary Disease

ENCORE: Study to Evaluate ALIS (Amikacin Liposome Inhalation Suspension) in Participants With Nontuberculous Mycobacterial Lung Infection Caused by Mycobacterium Avium Complex (clinicaltrials.gov) - P3 | N=425 | Active, not recruiting | Sponsor: Insmed Incorporated | Recruiting ➔ Active, not recruiting

Enrollment closed • Nontuberculous mycobacteria • Infectious Disease • Nontuberculous Mycobacterial Disease • Respiratory Diseases

Outcomes and characteristics of patients treated with nebulised amikacin liposome inhalation suspension (Arikayce®): report from a tertiary centre (BTS WM 2024) - "Am J Crit Care Med 2018; 198 (12):1559–1569. View this table: View inline View popup Abstract S44 Table 1 Comparison of responders vs. non-responders"

Clinical • Bronchiectasis • Inflammation • Non‐Cystic Fibrosis Bronchiectasis • Pulmonary Disease • Respiratory Diseases

LONGER USE OF AMIKACIN LIPOSOMAL INHALATION SUSPENSION DURING FIRST 12 MONTHS ASSOCIATED WITH LOWER RISK OF HOSPITALIZATIONS AND EMERGENCY ROOM VISITS: A CLAIMS ANALYSIS (CHEST 2024) - "PURPOSE: Amikacin liposome inhalation suspension (ALIS, Arikayce®) with multidrug regimen (MDR) is the only therapy approved for treatment of Mycobacterium avium complex lung disease (MACLD) in patients who do not achieve culture conversion (CC) after ≥6 consecutive months of MDR. Patients with longer use of ALIS use during the first 12 months following initiation were less likely to experience hospitalization or ER visits at follow-up than patients with shorter use. CLINICAL IMPLICATIONS: The health care resource utilization outcomes observed with longer ALIS use in this study is worth considering when tailoring treatment plans for patients with refractory MACLD. However, more work is needed to understand severity of disease and other confounding factors that may impact ALIS use and outcomes."

Clinical • Asthma • Cardiovascular • Chronic Obstructive Pulmonary Disease • Cough • Diabetes • Dysphonia • Immunology • Infectious Disease • Metabolic Disorders • Pneumonia • Pulmonary Disease • Respiratory Diseases